Patients and families with Huntington's disease (HD) as well as HD clinicians and researchers worldwide have been extremely interested in the recent exploration of potential surgical treatments for HD. Various surgical procedures have been proposed, and in some cases performed, including transplantation of human or pig fetal cells, and permanent destructive lesions of the brain (pallidotomy). The Huntington Study Group (HSG), a group of academically-based HD clinicians and researchers, has carefully and extensively monitored these developments with both hope and growing concern.
Based on a careful evaluation of all available information, we conclude that all potential surgical treatments are experimental and unproven regarding both safety and efficacy. We believe that the only responsible way for HD patients to receive any potential surgical treatment is in a well planned, well executed, carefully monitored controlled clinical trial designed to initially assess safety and, eventually, efficacy. Patients and families entering these trials must be informed of all available current information and should understand that no potential surgical treatment is known to be safe or effective.
Participation in surgical clinical trials will carry the risk of harm, potentially irreversible, as well as the possibility of improvement. Patients and families with HD should carefully consider participation in such trials and would benefit from consultation with HD caregivers knowledgeable about currently available experimental protocols. The following are a series of questions and answers that many HD patients and families may find useful in considering these decisions.
Why have clinical trials of surgery started if we do not know if the surgery is safe or effective?
For both experimental drug and surgical therapy trials, we rely on results from studies in animals, and from human experience with similar therapies to decide if there are enough reasons to try a new therapy. In the case of both transplantation and destructive brain lesions, many HD investigators and researchers believe there is enough reason to begin trials; however, some researchers do not agree. Further experiments continue in animals to help refine and guide new experimental surgical therapies.
How can patients and families tell if a clinical trial study is well designed?
The HD research community has not identified any single study design as preferred; however, there are essential elements in all well designed HD studies. One is that patients need to be informed about the study and the experimental nature of the surgery. A written informed consent document reviewed by an Institutional Review Board should be available for review and discussion. Also, patients must be evaluated in a structured manner by experienced HD clinicians for at least 6 months before surgery to adequately define HD symptoms. The same structured evaluations should continue for at least 2 years after surgery. The structured testing should include measures of movement, thinking and behavior. The study should allow for direct comparisons to patients who do not receive surgery or receive a different kind of surgery. Some patients involved in the trial may not initially get any surgery. This is the only well accepted way to measure the safety and efficacy of a new experimental treatment. The federal government prohibits charging patients for experimental drugs, and patients are usually not charged for the costs of experimental surgery.
How many studies have shown that the experimental surgery may help?
There are no published reports of well designed trials of surgery in HD. The few published reports of patients who have had surgery have received it in a setting that did not allow the safety or efficacy to be measured.
We strongly recommend that HD patients participate only in well designed and conducted clinical trials of any experimental treatment, surgery or drug. Patients and families with HD should understand that all current trials of potential surgical treatments are designed to measure safety, and that clinical trials designed to measure benefit are not currently available.
1/10/97 K. Kieburtz, K. Shannon for the HSG Executive Committee (I. Shoulson, J. Penney, D. Oakes, V. Hunt, K. Marder, J. Paulsen, M. Guttman, A. Young)